Monday, 9 November 2015

Gene editing: a therapy with masses of potential, but not only in treating cancer…

Last
week gene editing hit the news with the story of young leukaemia patient Layla
and a pioneering treatment at Great Ormond Street. Here Dr Anoushka de Almeida,
Head of Research at the Cystic Fibrosis Trust, talks about the science behind
the story, and the ground-breaking work being funded by the Trust into using
genetic editing for treating cystic fibrosis.

We have recently heard on the media of clinicians
at Great Ormond Street Hospital using the revolutionary technique of gene
editing to treat a little girl, Layla, diagnosed with an aggressive cancer of
the bone marrow. All other conventional treatments failed and this was Layla
and her family’s last hope – and it seems to have worked.

So what did they do to produce such a
miracle? Layla’s doctors teamed up with researchers at UCL, led by Prof Waseem
Qasim, who has developed a novel approach to gene editing in immune cells (T-cells).
This approach involves taking donated T-cells from a healthy person, modifying
the genome using gene editing to enable them to attack cancer cells, and then
introducing them into the body. Previously, these cells had only been tested in
mice, so Layla was the first human to receive them. The gene editing technique they used involved
using a pair of 'molecular scissors’, a kind known as TALEN proteins, to
‘switch off’ certain receptors, making sure that the modified T-cells leave the
healthy cells alone and only attack leukaemia cells. Genes were also edited out
to make the new cells ‘invisible’, so that they wouldn't be destroyed by other
leukaemia drugs.

It is still early to say whether Layla has
been completely cured. The pharma company Cellectis, who collaborated with
Qasim, plans to start full clinical trials early in 2016, the results of which
will hopefully confirm that this isn’t just a one-off. If they are successful,
this is a huge step forward for treating leukaemia and other cancers, as well
as other conditions…

This leads me on to talking about the
research that’s currently underway using gene editing in cystic fibrosis…

As we know, cystic fibrosis is caused when there are mutations in both copies of the
CFTR gene. Recent studies have shown that the most common CF-causing mutation,
F508del, can be corrected using a gene editing technique called ‘CRISPR/Cas9’ (a
different method to what they used for Layla – but principally the same). The
first step in this technique is to cut out the dodgy bit of genome using the
‘molecular scissor’ complex, and the second step is to repair it by using a
donor DNA molecule containing the correct, non-faulty sequence. Now, this all
works well in isolated cells outside the body, but the efficiency of the repair
is very low. Also, difficulty arises when delivering both the molecular scissor
complex and the donor DNA into the
correct place in the body.

A different approach is being investigated by Dr Patrick Harrison and colleagues at
University College Cork. This work, which the Cystic Fibrosis Trust is
co-funding with the Cystic Fibrosis Foundation in the US, involves focusing on a small group of three rare CF-causing mutations.
(These mutations basically disrupt the processing of the messenger RNA molecule
which is normally needed to make the CFTR protein in cells). In all three
cases, there is an extra sequence in
the genome which interferes with the normal processing. Dr Harrison’s team is exploring
using the gene editing approach simply to cut out this extra piece in the
genome region that causes the problem. This CRISPR ‘knock-out’ strategy is more
efficient than repair, and only requires delivery of just the molecular scissor
complex; no donor DNA is required.

Dr Harrison’s
group has teamed up with groups in Europe and the US to take this work further
so that they can determine if all this hard work eventually leads to the normal
CFTR protein being produced, resulting in normal functioning of the particular lung
cells in cystic fibrosis.

So, off the back of the break-through in
little Layla, gene editing has re-entered the limelight. It can be safely said that it is not only a promising
prospect for cancer such as leukaemia, as her case unquestioningly
demonstrates, but also for genetic conditions like cystic fibrosis. The Cystic
Fibrosis Trust is fully supportive of this therapy and sees its exciting
potential, and we would be keen to engage in further cutting-edge research in
this area of genetic therapies.

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